On February 24, 2021 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the fourth quarter of 2020 and recent business highlights (Press release, Ionis Pharmaceuticals, FEB 24, 2021, View Source [SID1234575529]).
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"Last year, we laid out a bold new vision for the Company and took important steps towards our goal of becoming one of the most successful biotechnology companies. Key to our vision is our strategy to maximize the value of our pipeline by commercializing our wholly owned medicines. Our acquisition of Akcea was an important step in building our commercial capabilities while enabling us to further strengthen our organization," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "Last year, we also advanced our late-stage pipeline and expanded the utility of our technology. Looking ahead, we expect data from multiple wholly owned programs in the first half of this year, followed by Phase 3 tofersen data in patients with SOD1-ALS in the second half. These key upcoming catalysts, together with our recent pipeline and technology achievements, position us well to have 12 or more products on the market in 2026. Importantly, we continue to have the financial strength to expand investment in our wholly owned pipeline and commercial capabilities to drive meaningful and increasing value for patients and shareholders."
2020 Summary Financial Results
Achieved 2020 financial guidance
$729 million in total revenues, with half from marketed products
$640 million of operating expenses on a non-GAAP basis(1) and $901 million on a GAAP basis, reflecting investments in Ionis’ wholly owned pipeline
Net income of $111 million on a non-GAAP basis(1) and a net loss of $451 million on a GAAP basis
Strong balance sheet with cash of $1.9 billion at year-end, enabling increasing investment in advancing the pipeline and technology while also preparing to commercialize the Company’s wholly owned medicines
2020 Marketed Products Highlights
SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
$2 billion in worldwide sales in 2020
More than 11,000 patients worldwide were on therapy at the end of the fourth quarter across post-marketing, expanded access and clinical trial settings
Enrollment began in the RESPOND study evaluating potential SPINRAZA benefit in SMA patients with a suboptimal clinical response to gene therapy
Enrollment began in the pivotal randomized treatment cohort of the DEVOTE study evaluating higher doses of SPINRAZA
TEGSEDI and WAYLIVRA: transformational medicines approved for the treatment of patients with severe rare diseases
Product sales increased more than 65 percent in 2020, compared to 2019
Generated growing revenues as major markets launched in 2020
Restructured European operations through a distribution agreement with Swedish Orphan Biovitrum AB (Sobi)
Q4 2020 and Recent Pipeline Highlights
Phase 3 Pipeline: growing pipeline positioned for 12 or more products on the market in 2026
Advanced IONIS-APOCIII-LRx into the Phase 3 BALANCE study in patients with FCS
Completed enrollment in the tofersen Phase 3 VALOR study in patients with SOD1-ALS
Mid-stage Pipeline: broad and advancing pipeline of potential first-in-class and/or best-in-class medicines
Advanced and expanded the IONIS-AGT-LRx development program
Reported IONIS-AGT-LRx positive topline Phase 2 results in patients with hypertension uncontrolled with two or three antihypertensive medications
Advanced ION904, the follow-on medicine targeting AGT, into Phase 1 development in healthy volunteers
Advanced vupanorsen into Phase 2b development with the initiation of the TRANSLATE-TIMI 70 dose-ranging study in statin-treated patients with dyslipidemia, resulting in a $75 million payment from Pfizer
Advanced ION449 (AZD8233), targeting PCSK9, into Phase 2b development in patients with dyslipidemia and AstraZeneca licensed ION455, a new investigational medicine for the treatment of nonalcoholic steatohepatitis (NASH), resulting in $50 million from AstraZeneca
Unlocked potential new pulmonary disease franchise with positive IONIS-ENAC-2.5Rx data
Reported positive healthy volunteer results supporting aerosol antisense medicine delivery to the lung
Completed dosing in the Phase 2 study in patients with cystic fibrosis
Advanced IONIS-ENAC-2.5Rx into Phase 2 development in patients with chronic obstructive pulmonary disease (COPD)
Highlighted IONIS-MAPTRx (BIIB080) Phase 1/2 study in patients with Alzheimer’s disease in which IONIS-MAPTRx was generally well tolerated and demonstrated durable, time and dose-dependent reductions in CSF tau protein
Upcoming 2021 Pipeline Catalysts
2020 Financial Results and 2021 Financial Guidance
"We achieved our 2020 financial guidance, even in the challenging COVID-19 pandemic environment. Moreover, in 2020 we made significant progress toward our goal of creating a stronger, more efficient company focused primarily on advancing our wholly owned medicines to the market. We acquired Akcea enabling us to retain full value from its rich portfolio. We also restructured our European operations. Together, these transactions unlocked substantial cost savings that we plan to reinvest to drive future revenue growth," said Elizabeth L. Hougen, chief financial officer of Ionis. "Our 2021 guidance reflects our new strategy to maximize the value of our wholly owned pipeline, focused primarily on commercializing our rare neurological and cardiometabolic disease programs. Our guidance also reflects the investments we are making in three key areas of our business – advancing and expanding our wholly owned pipeline, building commercial capabilities in support of our rich pipeline and broadening the reach of our technology. We can increase our investments in these areas while only modestly increasing our expenses because of the significant cost savings we realized from acquiring Akcea and restructuring our European operations. Importantly, with nearly $2 billion of cash at the end of last year, we remain well capitalized with the substantial financial resources to achieve our goals."
2021 Financial Guidance
Ionis’ full year 2021 financial guidance consists of the following components (on a non-GAAP basis)(1):
All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards and expenses related to the Akcea acquisition and restructured European operations and the related tax effects. Please refer to the section below titled "Financial Impacts of Akcea Acquisition and Restructured European Operations" for a breakdown of the costs specific to these transactions. Additionally, please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release.
Revenue
Financial Impacts of Akcea Acquisition and Restructured European Operations
In the fourth quarter of 2020, the Company’s non-GAAP amounts exclude the following expenses related to the Akcea acquisition and restructured European operations because the costs are not part of its normal ongoing operating activities. Refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release. (Amounts in millions):
As a result of the Akcea acquisition, Ionis and Akcea began reporting their federal taxes on a consolidated basis in the fourth quarter of 2020. The Company recorded a valuation allowance against all Ionis’ federal net deferred tax assets in the fourth quarter of 2020, due largely to Akcea’s history of losses and the expected impact of this on Ionis’ consolidated federal taxable income. The Company now maintains a valuation allowance against all its consolidated federal and state net deferred tax assets.
Operating Expenses
Ionis’ operating expenses for the year ended December 31, 2020 increased compared to 2019 driven by the Company’s investments in advancing the Phase 3 programs for IONIS-TTR-LRx, IONIS-APOCIII-LRx and other medicines in its wholly owned pipeline. Additionally, the Company incurred approximately $90 million in costs related to the Akcea acquisition and restructured European operations on a GAAP basis. The costs consisted of $31 million of severance, retention and other costs and $59 million of non-cash stock-based compensation expense for the acceleration of Akcea equity awards.
Net Loss Attributable to Noncontrolling Interest in Akcea
Prior to completing its acquisition of Akcea in October 2020, Ionis owned approximately 76 percent of Akcea. The line titled "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations reflects the portion of Akcea’s net income or loss attributable to the other owners of Akcea’s common stock. From mid-October 2020 through December 31, 2020, Ionis did not recognize any noncontrolling interest in Akcea on its statement of operations because it owned 100 percent of Akcea. Beginning in 2021, the Company will no longer have an adjustment for noncontrolling interest in Akcea.
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis recognized a net loss attributable to Ionis’ common stockholders for 2020 compared to net income in 2019 primarily due to higher revenue in 2019, including approximately $400 million in license fees Ionis earned from Pfizer and Novartis. Also contributing to Ionis’ net loss in 2020 was the non-cash adjustment of the valuation allowance Ionis recorded against its federal net deferred tax assets discussed above. Additionally, Ionis’ operating expenses increased in 2020 compared to the same period last year as described above.
Balance Sheet
Ionis ended 2020 with cash, cash equivalents and short-term investments of $1.9 billion, compared to $2.5 billion at December 31, 2019. In October 2020, Ionis used approximately $545 million of its cash for the Akcea acquisition.
Webcast
Today, at 9:00 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.